Data from the US and UK cystic fibrosis registries support disease modification by CFTR modulation with ivacaftor

Leona Bessonova, Nataliya Volkova, Mark Higgins, Leif Bengtsson, Simon Tian, Christopher Simard, Michael W. Konstan, Gregory S. Sawicki, Ase Sewall, Stephen Nyangoma, Alexander Elbert, Bruce C. Marshall, Diana Bilton

Research output: Contribution to journalArticlepeer-review

58 Scopus citations


Background Ivacaftor is the first cystic fibrosis transmembrane conductance regulator (CFTR) modulator demonstrating clinical benefit in patients with cystic fibrosis (CF). As ivacaftor is intended for chronic, lifelong use, understanding long-term effects is important for patients and healthcare providers. Objective This ongoing, observational, postapproval safety study evaluates clinical outcomes and disease progression in ivacaftor-treated patients using data from the US and the UK CF registries following commercial availability. Methods Annual analyses compare ivacaftor-treated and untreated matched comparator patients for: Risks of death, transplantation, hospitalisation, pulmonary exacerbation; prevalence of CF-related complications and microorganisms and lung function changes in a subset of patients who initiated ivacaftor in the first year of commercial availability. Results from the 2014 analyses (2 and 3 years following commercial availability in the UK and USA, respectively) are presented here. Results Analyses included 1256 ivacaftor-treated and 6200 comparator patients from the USA and 411 ivacaftor-treated and 2069 comparator patients from the UK. No new safety concerns were identified based on the evaluation of clinical outcomes included in the analyses. As part of safety evaluations, ivacaftor-treated US patients were observed to have significantly lower risks of death (0.6% vs 1.6%, p=0.0110), transplantation (0.2% vs 1.1%, p=0.0017), hospitalisation (27.5% vs 43.1%, p<0.0001) and pulmonary exacerbation (27.8% vs 43.3%, p<0.0001) relative to comparators; trends were similar in the UK. In both registries, ivacaftor-treated patients had a lower prevalence of CF-related complications and select microorganisms and had better preserved lung function. Conclusions While general limitations of observational research apply, analyses revealed favourable results for clinically important outcomes among ivacaftor-treated patients, adding to the growing body of literature supporting disease modification by CFTR modulation with ivacaftor.

Original languageEnglish (US)
Pages (from-to)731-740
Number of pages10
Issue number8
StatePublished - Aug 1 2018


  • cystic fibrosis
  • rare lung diseases
  • respiratory infection
  • systemic disease and lungs

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine


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